Price of PTC-124
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Treat Cystic Fibrosis and Duchenne and Becker Muscular Dystrophy (nmDMD/BMD).Orally bioavailable 284 D-1,2,4-oxadiazole.Promotes readthrough of premature translation termination codons. Targets genetic disorders caused by nonsense mutations.Shows no concomitant effects on normal termination or mRNA decay.